FDA Accepts Luspatercept BLA for Myelodysplastic Syndrome, Beta-thalassemia Indications


The FDA has accepted and set action dates for the luspatercept (Celgene and Acceleron) Biologic License Application.

Officials with the FDA have accepted Celgene’s Biologics License Application (BLA) for luspatercept, an investigational erythroid maturation agent, for myelodysplastic syndromes (MDS) and beta-thalassemia indications, according to a press release.

Celgene and Acceleron Pharma are seeking approval for the use of luspatercept in very low- to intermediate-risk MDS-associated anemia in adults who have ring sideroblasts and require red blood cell (RBC) transfusions. The FDA has set a Prescription Drug User Fee Act (PDUFA) date of April 4, 2020, for this indication.

Additionally, the agency granted Priority Review to luspatercept for the evaluation of the treatment in patients with beta-thalassemia-associated anemia who require RBC transfusions. A PDUFA date of December 4, 2019, has been set for this indication.

Acceptance of the BLA was based on safety and efficacy results from the phase 3 studies MEDALIST and BELIEVE. The studies evaluated lustapercept as a treatment for anemia associated with MDS and beta-thalassemia, respectively.


The MEDALIST trial included 229 adult patients with very low-, low-, or intermediate-risk MDS who were RBC transfusion dependent. All patients were either refractory or intolerant to prior erythropoiesis-stimulating agent (ESA) therapy or were ESA naïve with endogenous serum erythropoietin ≥200 U/L and had no prior treatment with disease modifying agents. Patients received either luspatercept 1 mg/kg or a placebo by subcutaneous injection once every 21 days. The median transfusion burden in both treatment arms was 5 RBC units/8 weeks, according to the study.


In the BELIEVE trial, luspatercept plus best supportive care (BSC) was compared with placebo plus BSC in 336 adults with beta-thalassemia patients who require regular RBC transfusions. Patients were randomized to receive either lupsatercept 1 mg/kg or a placebo by subcutaneous injection every 21 days for up to 48 weeks. Based on recommendation of an Independent Data Safety Monitoring Committee, crossover to the luspatercept treatment groups was allowed after unblinding. Luspatercept-treated patients will be followed for up to 3 years.

Additionally, a phase 3 trial in ESA-naïve, lower-risk patients with MDS, a phase 2 trial in non-transfusion-dependent beta-thalassemia, and a phase 2 trial in myelofibrosis are ongoing.

“The acceptance of the luspatercept filings and granting of the US priority review for beta-thalassemia represent another important step in delivering this novel therapy to patients in need,” Jay Backstrom, MD, chief medical officer for Celgene, said in a statement. “We believe that luspatercept can play a critical role in treating anemia associated with these serious blood diseases, and with these milestones achieved we look forward to working closely with the agency to move this therapy toward approval.”

Luspatercept is being jointly developed by Acceleron and Celgene.


Celgene Corporation and Acceleron Pharma Announce US FDA Accepts Luspatercept Biologics License Application in Myelodysplastic Syndromes and Beta-Thalassemia [news release]. Celgene. https://ir.celgene.com/press-releases/press-release-details/2019/Celgene-Corporation-and-Acceleron-Pharma-Announce-US-FDA-Accepts-Luspatercept-Biologics-License-Application-in-Myelodysplastic-Syndromes-and-Beta-Thalassemia/default.aspx. Accessed June 5, 2019.

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